CN104936606A - 糖原贮积病的基因疗法 - Google Patents

糖原贮积病的基因疗法 Download PDF

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Publication number
CN104936606A
CN104936606A CN201380054477.1A CN201380054477A CN104936606A CN 104936606 A CN104936606 A CN 104936606A CN 201380054477 A CN201380054477 A CN 201380054477A CN 104936606 A CN104936606 A CN 104936606A
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disease
methods
tfeb
gsd
muscle
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G·帕伦缇
A·巴拉比奥
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Fondazione Telethon
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Fondazione Telethon
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    • C12N2799/00—Uses of viruses
    • C12N2799/02—Uses of viruses as vector
    • C12N2799/021—Uses of viruses as vector for the expression of a heterologous nucleic acid
    • C12N2799/025—Uses of viruses as vector for the expression of a heterologous nucleic acid where the vector is derived from a parvovirus

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CN201380054477.1A 2012-10-17 2013-10-17 糖原贮积病的基因疗法 Pending CN104936606A (zh)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201261715187P 2012-10-17 2012-10-17
US61/715,187 2012-10-17
PCT/EP2013/003131 WO2014060109A1 (en) 2012-10-17 2013-10-17 Gene therapy for glycogen storage diseases

Publications (1)

Publication Number Publication Date
CN104936606A true CN104936606A (zh) 2015-09-23

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US (1) US20150273016A1 (pt)
EP (1) EP2908842A1 (pt)
JP (1) JP2015533368A (pt)
KR (1) KR20150081434A (pt)
CN (1) CN104936606A (pt)
AU (1) AU2013332061A1 (pt)
BR (1) BR112015008673A2 (pt)
CA (1) CA2888356A1 (pt)
WO (1) WO2014060109A1 (pt)

Cited By (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN110724203A (zh) * 2019-11-08 2020-01-24 中国人民解放军第四军医大学 一种促进tfeb核转位的短肽及基于其的线性短肽和其减轻脑缺血损伤的应用
CN110914419A (zh) * 2017-03-10 2020-03-24 吉尼松公司 糖原贮积病iii的治疗
CN113058041A (zh) * 2020-08-27 2021-07-02 华东师范大学 一种用于治疗庞贝氏病的产品

Families Citing this family (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
AU2017209189B2 (en) * 2016-01-19 2024-03-28 The Regents Of The University Of California Methods for the treatment of Danon disease and other disorders of autophagy
JP7048574B2 (ja) * 2017-03-10 2022-04-05 国立研究開発法人国立成育医療研究センター アンチセンスオリゴヌクレオチドおよび糖原病Ｉａ型予防または治療用組成物
JP6884268B2 (ja) 2018-03-09 2021-06-09 第一三共株式会社 糖原病Ｉａ型治療薬
WO2020014523A1 (en) 2018-07-12 2020-01-16 Rocket Pharmaceuticals, Ltd. Gene therapy vectors for treatment of danon disease
US20200385756A1 (en) * 2019-06-07 2020-12-10 Stc.Unm Optogenetic gene expression systems and methods
US20230048492A1 (en) * 2021-06-21 2023-02-16 The Brigham And Women`S Hospital, Inc. Adeno-Associated Viral (AAV) Vectors for Tissue-Targeted Expression of Therapeutic Genes

Family Cites Families (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN103458970A (zh) * 2011-03-07 2013-12-18 泰莱托恩基金会 Tfeb磷酸化抑制剂及其应用

Cited By (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN110914419A (zh) * 2017-03-10 2020-03-24 吉尼松公司 糖原贮积病iii的治疗
CN110724203A (zh) * 2019-11-08 2020-01-24 中国人民解放军第四军医大学 一种促进tfeb核转位的短肽及基于其的线性短肽和其减轻脑缺血损伤的应用
CN110724203B (zh) * 2019-11-08 2021-04-30 中国人民解放军第四军医大学 一种促进tfeb核转位的短肽及基于其的线性短肽和其减轻脑缺血损伤的应用
CN113058041A (zh) * 2020-08-27 2021-07-02 华东师范大学 一种用于治疗庞贝氏病的产品

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Publication number Publication date
US20150273016A1 (en) 2015-10-01
EP2908842A1 (en) 2015-08-26
WO2014060109A1 (en) 2014-04-24
KR20150081434A (ko) 2015-07-14
BR112015008673A2 (pt) 2017-09-26
AU2013332061A1 (en) 2015-05-07
JP2015533368A (ja) 2015-11-24
CA2888356A1 (en) 2014-04-24

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Application publication date: 20150923